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Safety and Efficacy of Inhaled Iloprost in Pediatric Patients With Pulmonary Arterial Hypertension

Primary Purpose

Pulmonary Arterial Hypertension

Status

Withdrawn

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

Iloprost Inhalation Solution (Ventavis)

About this trial

This is an interventional treatment trial for Pulmonary Arterial Hypertension focused on measuring PAH, Pediatric, Pulmonary Arterial Hypertension

Eligibility Criteria

3 Years - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Male or female, age 3-18 years
Diagnosis of PAH due to IPAH, connective tissue disease, or congenital heart disease
Pulmonary arterial hypertension confirmed by a cardiac catheterization, with mPAP > 25 mmHg at rest, PCWP or LVEDP ≤ 15 mmHg, and PVR ≥ 240 dynes/sec/cm-5 (ie, ≥ 3.0 units m2)
NYHA/WHO functional Class II, III, or IV
Have had a 6-minute walk test (6-MWT) performed within the 3 months preceding the screening visit (if ≥ 8 years of age)
Have had cardiopulmonary exercise testing (CPET) performed within the 3 months preceding the screening visit (if ≥ 8 years of age and > 130 cms in height. CPET testing will be conducted on NYHA/WHO Class IV patients at the discretion of the Principal Investigator)
If receiving therapy for PAH, on a stable dose and regimen for at least 3 months prior to the screening visit
If receiving digoxin, diuretic, or oxygen therapy, on a stable dose and regimen for at least 30 days preceding the screening visit
Must demonstrate the ability to appropriately use the nebulizer device as part of the screening process

Exclusion Criteria:

Prior use of prostacyclins or prostacyclin analogues within 3 months prior to screening
Portal hypertension or clinically relevant hepatic disease
Eisenmenger syndrome with resting SpO2 <88% on room air
Clinically relevant asthma or other chronic lung disease, such as bronchopulmonary dysplasia, cystic fibrosis, or repaired or unrepaired congenital diaphragmatic hernia
Diagnosis or current evidence of active lung infection or clinically relevant hemoptysis
Left-sided heart disease, including valvular disease or heart failure
Initiation or dosage adjustment of PAH-specific therapy within 3 months prior to screening
Initiation of treatment with digoxin, diuretics, anticoagulation, or oxygen therapy within 30 days prior to screening

Sites / Locations

The Children's Hospital
Columbia University Medical Center

Outcomes

Primary Outcome Measures

Change from baseline in 6-minute walk distance measured post-inhalation (for patients ≥ 8 years old) at Week 8 and Week 16

Change from baseline in Borg Dyspnea Scale measured following 6-MWT (for patients ≥ 8 years old) at Week 8 and Week 16

Change from baseline in NYHA/WHO functional class at Week 8 and Week 16

Change from baseline in hemodynamic parameters measured at Week 16, and in patients who are able to undergo iloprost inhalation while consciously sedated, hemodynamic parameters measured prior to dosing and 15-30 minutes after dosing

Time to clinical worsening, defined as any one of the following: death due to underlying PAH, transplantation, atrial septostomy, hospitalization for PAH or right heart failure, >15% worsening in 6-minute walk distance in concert with a ≥ 1 class deteri

Change from baseline in Child Health Questionnaire (CHQ-28) at Week 8 and Week 16

Change from baseline in exercise capacity, as measured by post-inhalation CPET, at Week 8 and Week 16 (if ≥ 8 years old and > 130 cms in height. CPET testing will be conducted on NYHA/WHO Class IV patients at the discretion of the Principal Investigat

Secondary Outcome Measures

Full Information

NCT ID

NCT00453414

First Posted

March 27, 2007

Last Updated

February 12, 2010

Sponsor

Actelion

1. Study Identification

Unique Protocol Identification Number

NCT00453414

Brief Title

Safety and Efficacy of Inhaled Iloprost in Pediatric Patients With Pulmonary Arterial Hypertension

Official Title

Safety and Efficacy of Inhaled Iloprost in Pediatric Patients With Pulmonary Arterial Hypertension

Study Type

Interventional

2. Study Status

Record Verification Date

February 2010

Overall Recruitment Status

Withdrawn

Why Stopped

Withdrawn due to lack of enrollment

Study Start Date

July 2006 (undefined)

Primary Completion Date

October 2006 (Anticipated)

Study Completion Date

October 2006 (Anticipated)

3. Sponsor/Collaborators

Name of the Sponsor

Actelion

4. Oversight

5. Study Description

Brief Summary

The purpose of this study is to evaluate the safety and effectiveness of iloprost for PAH in children who are between the ages of 3 and 18 years old.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Pulmonary Arterial Hypertension

Keywords

PAH, Pediatric, Pulmonary Arterial Hypertension

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Intervention Type

Drug

Intervention Name(s)

Iloprost Inhalation Solution (Ventavis)

Primary Outcome Measure Information:

Title

Change from baseline in 6-minute walk distance measured post-inhalation (for patients ≥ 8 years old) at Week 8 and Week 16

Title

Change from baseline in Borg Dyspnea Scale measured following 6-MWT (for patients ≥ 8 years old) at Week 8 and Week 16

Title

Change from baseline in NYHA/WHO functional class at Week 8 and Week 16

Title

Change from baseline in Child Health Questionnaire (CHQ-28) at Week 8 and Week 16

Title

10. Eligibility

Sex

All

Minimum Age & Unit of Time

3 Years

Maximum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Male or female, age 3-18 years Diagnosis of PAH due to IPAH, connective tissue disease, or congenital heart disease Pulmonary arterial hypertension confirmed by a cardiac catheterization, with mPAP > 25 mmHg at rest, PCWP or LVEDP ≤ 15 mmHg, and PVR ≥ 240 dynes/sec/cm-5 (ie, ≥ 3.0 units m2) NYHA/WHO functional Class II, III, or IV Have had a 6-minute walk test (6-MWT) performed within the 3 months preceding the screening visit (if ≥ 8 years of age) Have had cardiopulmonary exercise testing (CPET) performed within the 3 months preceding the screening visit (if ≥ 8 years of age and > 130 cms in height. CPET testing will be conducted on NYHA/WHO Class IV patients at the discretion of the Principal Investigator) If receiving therapy for PAH, on a stable dose and regimen for at least 3 months prior to the screening visit If receiving digoxin, diuretic, or oxygen therapy, on a stable dose and regimen for at least 30 days preceding the screening visit Must demonstrate the ability to appropriately use the nebulizer device as part of the screening process Exclusion Criteria: Prior use of prostacyclins or prostacyclin analogues within 3 months prior to screening Portal hypertension or clinically relevant hepatic disease Eisenmenger syndrome with resting SpO2 <88% on room air Clinically relevant asthma or other chronic lung disease, such as bronchopulmonary dysplasia, cystic fibrosis, or repaired or unrepaired congenital diaphragmatic hernia Diagnosis or current evidence of active lung infection or clinically relevant hemoptysis Left-sided heart disease, including valvular disease or heart failure Initiation or dosage adjustment of PAH-specific therapy within 3 months prior to screening Initiation of treatment with digoxin, diuretics, anticoagulation, or oxygen therapy within 30 days prior to screening

Facility Information:

Facility Name

The Children's Hospital

City

Denver

State/Province

Colorado

ZIP/Postal Code

80218

Country

United States

Facility Name

Columbia University Medical Center

City

New York

State/Province

New York

ZIP/Postal Code

10032

Country

United States

12. IPD Sharing Statement

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Safety and Efficacy of Inhaled Iloprost in Pediatric Patients With Pulmonary Arterial Hypertension

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